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PTC Therapeutics Provides a Corporate Update

October 28, 2021

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2021.

“The stellar growth of our DMD franchise is remarkable.” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “I continue to be impressed by the team’s performance. The commercial performance, along with the milestones achieved in Brazil, and the continued advancements in our pipeline, with initiating a fifth registration-directed trial, allows us continued value creation for all of our stakeholders.”

Key Third Quarter and Other Corporate Updates:

  • The Duchenne muscular dystrophy (DMD) franchise grew 39% over the third quarter of 2020, demonstrating PTC’s continued strong commercial performance. Quarterly net product revenue for the franchise was $114 million in the third quarter of 2021.
    • Translarna™ (ataluren) revenue growth was driven by geographic expansion and new patient identification.
    • Emflaza® (deflazacort) revenue growth was driven primarily by new patient starts and maintained high compliance.
  • Evrysdi™(risdiplam) continues to show strong global uptake. The first commercial sale of Evrysdi™ in Japan occurred in August 2021, resulting in a $10 million milestone payment from Roche to PTC with $325 million in sales-based milestones remaining. Evrysdi™ is a product of a collaboration between PTC, Roche and the SMA Foundation.
  • Waylivra™ (volanesorsen) was approved by the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), as the first treatment for familial chylomicronemia syndrome (FCS) in Brazil.
  • Tegsedi® (inotersen) has successfully received Category 1 classification from CMED (Drug Market Regulation Chamber) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than current standards of care and allows for pricing in line with international markets.

Third Quarter Clinical Updates:

  • The APHENITY registration-directed Phase 3 trial for PTC923 for phenylketonuria (PKU) has been initiated with results expected by the end of 2022.
  • PTC continues to make progress in three additional ongoing registration-directed clinical studies:
    • The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures with results anticipated in the third quarter of 2022.
    • The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia with results anticipated in 2023.
    • Enrollment in the FITE19 Phase 2/3 emvododstat trial in patients with COVID-19 is expected to be completed by year end 2021.
  • The Phase 1 healthy volunteer study of PTC518 for Huntington’s disease met the objectives in the third quarter of this year. The Phase 2 study in patients with Huntington’s disease is expected to be initiated by the end of 2021.
  • The Committee for Medicinal Products for Human Use (CHMP) opinion on the PTC-AADC gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency is expected in the fourth quarter of 2021.
  • For the Biologics License Application (BLA) for AADC deficiency, PTC expects to submit the BLA in the first quarter of 2022.

Third Quarter 2021 Financial Highlights:

  • Total revenues were $138.7 million for the third quarter of 2021, compared to total revenues of $118.4 million for the third quarter of 2020.
  • Total revenue includes net product revenue across the commercial portfolio of $115.6 million and royalty and collaboration revenue of $23.1 million for the third quarter of 2021, compared to net product revenues of $82.7 million and royalty and collaboration revenues of $35.7 million for the third quarter of 2020.
  • Translarna net product revenues were $67.2 million for the third quarter of 2021, compared to $43.4 million for the third quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion.
  • Emflaza net product revenues were $47.1 million for the third quarter of 2021, compared to $38.5 million for the third quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
  • Roche reported Evrysdi 2021 year to date sales of approximately CHF 396 million, resulting in year-to-date royalty revenue of $33.3 million to PTC. During the third quarter of 2021, the first commercial sale of Evrysdi in Japan triggered a $10 million milestone payment from Roche to PTC, which was reported as collaboration revenue.
  • U.S. GAAP (generally accepted accounting principles) research and development (R&D) expenses were $130.8 million for the third quarter of 2021, compared to $93.0 million for the third quarter of 2020. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
  • Non-GAAP R&D expenses were $117.8 million for the third quarter of 2021, excluding $13.0 million in non-cash stock-based compensation expense, compared to $83.8 million for the third quarter of 2020, excluding $9.2 million in non-cash stock-based compensation expense.
  • GAAP selling, general and administrative (SG&A) expenses were $69.3 million for the third quarter of 2021, compared to $57.8 million for the third quarter of 2020. The increase reflects our continued investment to support commercial activities including expanding our commercial portfolio.
  • Non-GAAP SG&A expenses were $56.4 million for the third quarter of 2021, excluding $12.8 million in non-cash stock-based compensation expense, compared to $50.3 million for the third quarter of 2020, excluding $7.6 million in non-cash stock-based compensation expense.
  • Change in the fair value of deferred and contingent consideration was $10.8 million for the third quarter of 2021, compared to $8.4 million for the third quarter of 2020. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
  • Net loss was $133.6 million for the third quarter of 2021, compared to net loss of $69.7 million for the third quarter of 2020.
  • Cash, cash equivalents and marketable securities was $867.9 million at September 30, 2021, compared to $1.1 billion at December 31, 2020.
  • Shares issued and outstanding as of September 30, 2021 were 70,665,010.

PTC Updates Full Year 2021 Guidance as Follows:

  • PTC now anticipates net product revenues for the DMD franchise for the full year 2021 to be between $400 and $420 million from previous guidance of $370 and $390 million.
  • PTC now anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $815 and $835 million from previous guidance of $825 and $855 million.
  • PTC now anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $715 and $735 million, excluding estimated non-cash, stock-based compensation expense of $100 million, from previous guidance of $725 and $755 million.

About PTC Therapeutics, Inc.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The Company’s strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.